Developing Therapies for Diseases
of Misfolded Proteins
BioPartnering Conference
May 23-24, 2011
11 Deer Park Drive, Suite 103
Monmouth Junction, New Jersey 08852
www.exsar.com
ExSAR INTRODUCTION
Corporate Snapshot
¾ ExSAR Corporation is a small, privately-owned biotechnology company that was
founded in 1997, incorporated in Delaware, and has a current headcount of 7
employees all based in New Jersey.
¾ ExSAR is best known for our proprietary mass spectrometry-based platform
technology known as Hydrogen/Deuterium Exchange (aka H/D-Exchange), which
we offer to clients on a fee-for-service basis.
¾ The Company leveraged its H/D-Exchange platform to discover and develop
dr gs to treat protein folding disorders in 2005.
drugs
2005
¾ ExSAR has identified 2 small molecule compounds to treat diseases of misfolded
proteins, specifically Gaucher disease and Tay-Sachs disease. It is worth noting
that Tay-Sachs disease currently has NO approved therapy or treatment available.
available
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EXSAR’S THERAPEUTIC TARGETS OF INTEREST
Many Neurodegenerative, Metabolic and Inflammatory Diseases Caused by Misfolding,
Degradation and/or Aggregation of Proteins
h Combined market >$15 billion, e.g. Parkinson, ALS, Diabetes
h Our therapeutic area of interest is in diseases caused by misfolded proteins, and
more specifically a smaller subset known as Lysosomal Storage Disorders (LSDs)
h Common LSDs include Fabry, Gaucher, Pompe, Tay-Sachs, and Sandhoff
Diseases
h Both Tay-Sachs and Gaucher are classified as Orphan diseases as each affect a
very small group of individuals. Tay-Sachs & Sandhoff (which is closely related)
have a disease prevalence of ~1,500 patients worldwide; Gaucher, the most
common LSD, has a disease prevalence of ~11,000 patients worldwide
h Recent studies have shown a potential link between Gaucher disease and
Parkinson’s disease
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COMPETITION & INTELLECTUAL PROPERTY
ExSAR’s key value drivers
¾
Currently
C
tl no effective
ff ti therapy
th
or treatment
t
t
t exists
i t for
f Tay
T Sachs
S h disease,
di
so
there is no competition in this space.
¾
There is competition in the Gaucher disease area comprised of a handful of
companies most notably is Genzyme Corporation - with their enzyme
companies,
replacement therapy (ERT) drug Cerezyme ®.
¾
However, the mechanism of action of EXR-202 is notably different and acts as
a pharmacological chaperone to help in the protein refolding that is required.
¾
With regards to Cerezyme®, due to the high costs associated with using this
drug (~$200K per patient/per year for life), a small orally-delivered
pharmaceutical chaperone,
p
p
, such as EXR-202,, would be a cost effective and
hence attractive alternative to ERT, which is the current standard of care.
¾
ExSAR currently has 3 patents pending for the use of these compounds in
their respective markets.
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BUILDING A PIPELINE THROUGH COLLABORATIONS
Developed Relationships with Leading Institutions
h Tay-Sachs Disease and Gaucher Disease
ƒ Source of ExSAR’s license for EXR-101 and EXR-202 and site of preclinical
work completed by Dr. Don Mahuran of Hospital for Sick Children, Toronto
ƒ ExSAR helped fund a small Open Label Phase I/II of EXR
EXR-101
101 on Tay
Tay- Sachs
completed by Dr. Joe Clarke at Hospital for Sick Children in Toronto in 2010
ƒ ExSAR sponsored a small Phase I/II trial of EXR-202 on Gaucher completed
by Dr. Ari Zimran at Shaare Zedek Medical Center in Israel in 2010
h Familial Amyotrophic Lateral Sclerosis (ALS)
ƒ Collaborative research agreement with Dr.
Dr Jeffrey Agar of Brandeis University
to identify and validate inhibitors of superdioxide dismutase (SOD1)
aggregation
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KEY PERSONNEL
Experienced Leadership Team
Robert F. Johnston
President & CEO
Pharmos, Sepracor, Cytogen, I-STAT, Genex , Envirogen
Charles Cantor, PhD
Board Member
CSO & Chairman, Sequenom
Myra Williams, PhD
Board Member
former CEO Molecular Applications Group
F Raymond Salemme,
F.
Salemme PhD
Board Member
CEO, Redpoint Bio & founder 3D Pharma
Robert Towarnicki
Board Member
President & CEO, Makefield Therapeutics
S i tifi Ad
Scientific
Advisory
i
B
Board
d
William DeGrado, PhD
University of Pennsylvania
Edwin H. Kolodny, MD
NYU School of Medicine
Arnold Levine,, PhD
Institute for Advanced Study
y
Walter Englander, PhD
University of Pennsylvania
Patrick Griffin, PhD
The Scripps Research Institute
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EXR-202: GAUCHER OPPORTUNITY
Worldwide Prevalence of Gaucher Patients is Approximately 11,000
h ExSAR anticipates charging a price of $60,000
h Annual peak sales are estimated to be $100 - $130 million
Potential Annual Revenue Based on Market
Penetration
(USD Millio
ons)
$200
$160
$120
$80
$40
$0
1%
3%
7%
10%
15%
18%
25%
Market Penetration (%)
$60 000 Annual
$60,000
A
l Price
P i
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EXR-101: TAY SACHS OPPORTUNITY
Worldwide Prevalence of LOTS and Sandhoff Patients is Approximately 1,500
h ExSAR anticipates charging a price of $60,000 representing a $90 million
market
k t opportunity
t it worldwide
ld id
h Annual peak sales are estimated to be $30 - $40 million
P t ti l Annual
Potential
A
l Revenue
R
Based
B
d on Market
M k t
Penetration
(U
USD Millions)
$60
$50
$40
$30
$20
$10
$0
15%
25%
35%
45%
55%
65%
75%
Market Penetration (%)
$60,000 Annual Price
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REVENUE PROJECTIONS
Figures based on annual therapy costs of $60K for each drug
EXR 202
EXR-202
Scenario 1
Gaucher (WW)
% Registered
% Penetration
P ti t on ExSAR-202
Patients
E SAR 202
Annual Therapy Cost ($)
Annual Revenue ($)
EXR-101
Scenario 1
LOTS/SD Prevalence (WW)
% Registered
% Penetration
Patients on ExSAR-101
Annual Therapy Cost ($)
Annual Revenue ($)
Year 1
Year 2
Year 3
Year 4
11,000
11,220
11,444
11,673
70%
71%
72%
73%
1%
3%
7%
10%
77
239
577
852
60,000
60,000
60,000
60,000
4,620,000 14,339,160 34,607,866 51,129,001
Year 5
11,907
74%
15%
1 322
1,322
60,000
79,298,980
Year 1
Year 2
Year 3
Year 4
Year 5
1,500
1,530
1,561
1,592
1,624
50%
51%
52%
53%
54%
20%
%
30%
%
40%
%
50%
%
53%
%
150
234
325
422
460
60,000
60,000
60,000
60,000
60,000
9,000,000 14,045,400 19,476,288 25,309,811 27,618,257
Year 6
12,145
75%
18%
1 640
1,640
60,000
98,373,600
Year 6
1,656
55%
55%
%
501
60,000
30,058,600
FUNDING MILESTONES
$15 Million Funding
H1 ‘12
EXR-101
IND
H2 ‘12
H1 ‘13
P1
H2 ‘13
H1 ‘14
H2 ‘14
P2
$5.2 Million
EXR 202
EXR-202
IND
P1
P2
$5.1 Million
G&A/Research/
Service Business
Expenses
$1.9 Million
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FUNDING
¾
Raised approximately $12 million to date, with $11 million coming directly from
ExSAR’s President and CEO, Robert Johnston.
¾
Received Grant from FDA (DHHS) for Clinical Studies of Safety and Effectiveness
of Orphan Products for $400K in 2008
¾
ExSAR is seeking $15 million, which will enable us to initiate and complete more
extensive Phase I/II clinical trials, and to identify and in-license additional
compounds.
¾
We will be looking for corporate partnerships to conduct the pivotal Phase III
trials and commercialization.
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SUMMARY
¾
ExSAR is supported by a strong, experienced leadership team of executives,
di
directors
t
and
d scientific
i tifi advisors.
d i
¾
ExSAR has 2 small molecule compounds: EXR-101 and EXR 202, both targeting
orphan indications, one of which currently has NO effective treatment or therapy.
¾
Both compounds have a well-established safety profile in humans for other
indications, and each is likely to provide 7 to 10 years of market exclusivity in
both the US and Europe respectively, upon being granted Orphan Designation by
the regulatory agencies. ExSAR has already filed an IND for EXR-101.
EXR 101.
¾
We are asking for $15 million in funding to support additional clinical trials of
both EXR-101 and EXR-202.
¾
ExSAR is interested in exploring corporate partnerships in order to conduct
Phase III trials and commercialization of these drugs.
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Thanks to
& thank you all for your attention!
Robert F. Johnston President & CEO
[email protected]
Dawne Miller Head, Business Development [email protected]
www.exsar.com
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